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Lysogene S.A.. (2/2/17). "Press Release: Lysogene Receives Orphan Drug Designation from FDA for LYS-GM101 for Treatment of GM1 Gangliosidosis". Paris.

Region Region United States (USA)
Organisations Organisation Lysogene S.A. (Euronext Paris: LYS)
  Organisation 2 FDA (US Food and Drug Administration)
  Group United States (govt)
Products Product LYS-GM101 (gene therapy for GM1 Gangliosidosis)
  Product 2 DRUGS, ORPHAN
Persons Person Aiach, Karen (Lysogene 201206 CEO + Founder)
  Person 2 Ankri, Sarah (Lysogene 201710 VP Finance before Theraclion 201508 CFO + Board Director of Biotech France)

Lysogene (Paris:LYS), a leading, clinical-stage biotechnology company specializing in gene therapy for rare central nervous system diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to LYS-GM101, the company’s gene therapy drug candidate for treatment of GM1 Gangliosidosis (“GM1”).

“FDA Orphan Drug Designation for LYS-GM101 is an important regulatory milestone, which will facilitate clinical development of LYS-GM101. It is good news for patients that we can further study this therapy as this is a severe neurodegenerative disease with unmet medical need,” stated Karen Aiach, Founder and Chief Executive Officer of Lysogene. “We look forward to starting our upcoming phase I/II clinical trial (LYS-GM101) by the end of the first half of 2018, in line with the timetable previously announced.”

LYS-GM101 is designed to replace a defective gene in the cells of GM1 patients, in order to allow for production of the functional enzyme and to prevent the progressive nature of the neurological damage caused by GM1 in humans.

About the Orphan Drug Designation

Orphan Drug Designation is granted by the FDA to novel therapeutics for diseases or conditions affecting fewer than 200,000 patients in the U.S. or greater than 200,000 patients if there is no reasonable expectation that the production cost of the drug will be covered by its sales. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as, in some cases, tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About GM1

GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-galactosidase (ßgal) enzyme. The resulting enzymatic deficiency leads to accumulation of GM1-ganglioside in cells. Clinical presentation is mainly neurological with rapidly progressive impairment (motor, cognitive and behavioral) leading to premature death, mostly in early childhood. It is a devastating disease for patients and families. There is currently, to the best of the Company’s knowledge, no disease modifying treatment available.

About Lysogene :


Sarah Ankri, +33 (0)1 41 43 03 90
VP Finance

Record changed: 2023-06-05


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